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Phase 3 Gliofocus Study Drug, Niraparib, Granted Orphan Drug Designation by U.S. FDA for Treatment of Malignant Glioma, including Glioblastoma
- With over 250,000 new cases annually, and a 5-year survival rate of less than 5%, glioblastoma (GBM) is both the most common and lethal brain cancer in adults.
- The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to niraparib for the treatment of malignant glioma, including GBM.
- The FDA grants ODD to encourage the advancement, research, and development of new medicines for rare diseases with few treatment options.
- The Ivy Brain Tumor Center at Barrow Neurological Institute, sponsor of the Phase 3 Gliofocus Study, is evaluating niraparib versus temozolomide in newly diagnosed MGMT-unmethylated GBM.
The U.S. FDA has granted ODD to niraparib for the treatment of malignant glioma, including GBM. The designation is supported by the clinical trial interim data reported by the Ivy Brain Tumor Center at Barrow Neurological Institute. ODD is a special status granted by the FDA to medicines intended to treat, diagnose, or prevent rare diseases. This designation provides incentives such as regulatory support, tax credits, and potential market exclusivity to accelerate the development of treatments for patients with rare and life-threatening conditions.
“Niraparib receiving ODD is a pivotal moment in our singular goal to discover new treatments for GBM and other incurable brain tumors,” says Dr. Nader Sanai, director of the Ivy Brain Tumor Center and study chair. “This designation from the FDA not only recognizes the dire need for more effective therapies but underscores the promising results we observed in our Hybrid Phase 0 Study of niraparib. For patients, families, and caregivers, it brings hope during the most difficult moment of their lives.”
The Ivy Center’s Phase 3 Gliofocus Study, supported by GSK, compares niraparib monotherapy to the standard of care, temozolomide, in adult patients with newly diagnosed GBM tumors that lack a key chemical modification, called MGMT unmethylated.
GBM remains the most common and aggressive adult brain cancer, with limited advances within the past two decades. In approximately 60% of GBM tumors, the MGMT promoter is unmethylated,1 making the tumors resistant to temozolomide. The historical median overall survival for patients in this category is just 12.7 months.2,3 Positive interim data from the Ivy Center’s Phase 0/2 clinical trial of niraparib in GBM provided the foundation to advance the drug to a Phase 3 study. In that trial, niraparib achieved drug concentrations and target modulation in excess of any other studied PARP inhibitor. No new safety signals were identified.
Patients and care partners can learn more about Gliofocus enrollment by visiting Gliofocus.org and NCT06388733.
The Ivy Center is home to the largest Phase 0 drug development program for brain cancer worldwide. Click here for a complete list of Ivy Center clinical trials.
About Niraparib
The drug, niraparib, is an oral, once-daily PARP inhibitor with current indications in first-line and recurrent maintenance for advanced ovarian cancer. It is currently approved under the brand name Zejula for the treatment of ovarian cancer. The Gliofocus study is sponsored by the Ivy Brain Tumor Center with funding and collaboration from GSK.
About Ivy Brain Tumor Center
Ivy Brain Tumor Center at Barrow Neurological Institute in Phoenix, AZ, is a tertiary care and nonprofit translational research program that employs bold, early-phase clinical trial strategies to identify new treatments for aggressive brain tumors, including GBM. Our leading experts in neurosurgical oncology, neuro-oncology, radiation oncology, neuroradiology, neuropathology and neuroscience nursing treat more patients annually than any other brain tumor center in the United States. The Ivy Center’s Phase 0 clinical trials program is the largest in the world and enables personalized care in a fraction of the time and cost associated with traditional drug development. In addition, unlike conventional clinical trials focusing on single drugs, the Ivy Center’s accelerated program tests therapeutic combinations matched to individual patients. We leave no stone unturned in the pursuit of hope and healing. Learn more at IvyBrainTumorCenter.org. Follow the Ivy Brain Tumor Center on Facebook, Instagram, Twitter, and LinkedIn.